Quantifying the Altruism Value for a Rare Pediatric Disease: Duchenne Muscular Dystrophy

That is the title of a paper recently published in AJMC with co-authors Suhail Thahir, Alexa Klimchak, Ivana Audhya, Lauren Sedita, and John A. Romley. The abstract is below. Objectives: To quantify the magnitude of altruism value as applied to a hypothetical new treatment for a rare, severe pediatric disease: Duchenne muscular dystrophy (DMD). Study Design: Prospective…

HTA and cell and gene therapy

A paper by Drummond et al. (2023) examines how health technology assessment bodies deal with the challenge of value assessment for cell and gene therapies (also known as Advanced Therapy Medicinal Products (ATMPs) in Europe). To do this the authors: …undertook i) a targeted review of the literature on the clinical and economic evidence needs…